Medications

Neuromyelitis optica spectrum disorder (NMOSD) is a rare neuroinflammatory condition that may affect the eyes, spinal cord, and brain. Drug treatment for NMOSD has improved over recent years, and research continues to find more options for this condition.¹

Drug treatment for NMOSD can be described as either acute (immediate or short-term) or preventative. Acute drugs are used to limit symptoms of acute NMOSD attacks when they start. Preventive drugs are used to prevent the damaging future attacks of the disease from happening.^2,3

Understanding these drugs, how are they used in NMOSD, and what additional options may be available can help you get the right treatment.

Acute drugs

These are prescribed to treat inflammation and symptoms related to acute attacks of NMOSD.³

Steroids

Steroids (corticosteroids) are strong anti-inflammatory drugs. Your body makes steroids normally. When given in doses larger than what your body typically makes, steroids quickly decrease inflammation.⁴

High-dose steroids are currently the only type of drug used in an acute attack to specifically treat the inflammation from NMOSD. These drugs can help treat vision loss due to optic neuritis (inflammation of the nerve fibers in the eye) and neurologic symptoms due to transverse myelitis (inflammation of the spinal cord) or inflammation in the brain. Methylprednisolone is the most common IV steroid given to those with NMOSD.^1,3,5

Preventative drugs

Prevention of NMOSD attacks is vital. Each attack can lead to increased damage to the brain, eyes, and spinal cord. The goal of preventative treatment is to decrease the number and severity of future attacks. Until recently, preventive drugs approved by the US Food and Drug Administration (FDA) were not available to those with NMOSD.^6,7

In NMOSD, the antibody that attacks a protein called aquaporin-4 (AQP4) can result in damage to nerves and their protective coating in the optic nerve, spinal cord, and brain.⁸

As of 2024, the FDA has approved 4 drugs for the treatment of those with AQP4 antibody (AQP4-IgG) NMOSD. These drugs are known as biologics and consist of antibodies that are produced from living cells.^2,7

Soliris^® (eculizumab)

Soliris® is a laboratory-made antibody used to decrease the risk of an NMOSD attack. Soliris is an antibody that targets and inhibits the complement pathway. Activation of the complement pathway plays a key role in the development of NMOSD and resulting nerve damage. Soliris can increase the risk of certain bacterial infections. The drug is given by intravenous (IV) infusion, usually every 5 weeks.^9,10

Uplizna™ (inebilizumab)

Uplizna™ works by lowering the number of your body’s B cells, which are part of your immune response. Your B cells create antibodies, including the anti-AQP4 antibodies in NMOSD. With fewer of these cells, the immune system of those with NMOSD may not react as strongly to certain infections. Uplizna is given twice a year by IV infusion.^11,12

Enspryng™ (satralizumab)

Enspryng™ is the first approved treatment in NMOSD that was designed to block interleukin-6 (IL-6). IL-6 is a protein made by cells in the body and plays a role in the inflammation that occurs in NMOSD. Enspryng blocks IL-6 from attaching to the cell surface.^13,14

Enspryng is given by self-injection once per month. If your doctor prescribes this drug, you will receive injection training on the safe and correct way to inject it. Enspryng increases the risk of certain infections.^13,14

Ultomiris^® (ravulizumab)

Ultomiris® is the most recently FDA-approved NMOSD treatment. Ultomiris is an antibody that targets and inhibits the complement pathway, similar to Soliris. Ultomiris can also increase the risk of certain bacterial infections. The drug is given by IV infusion, usually every 8 weeks.¹⁵

Other preventative drugs

Off-label uses may be common in rare diseases where not many drugs are used to treat a condition. If you and your doctor decide on off-label use, it is important to understand that the FDA has not yet determined the drug is safe and effective when used the way you are taking it. However, this does not mean your use of the drug is unsafe. Often, it just means there has not been a large, official clinical trial of this specific drug for this specific rare disease. Talk to your doctor about your treatment questions and needs with off-label drugs for NMOSD.¹⁶

Some off-label and unapproved drugs commonly used to treat NMOSD include:^17-23

• Rituxan^® (rituximab), a laboratory-made antibody that decreases B cells
• Mitoxantrone, an anti-cancer drug
• Campath^® and Lemtrada^® (alemtuzumab), which are laboratory-made antibodies
• Imuran^® and Azasan^® (azathioprine), which are drugs that decrease the immune response
• CellCept^® (mycophenolate mofetil), a drug that decreases the immune response
• Trexall^®, Otrexup^®, and Rasuvo^® (methotrexate), which are disease-modifying antirheumatic drugs (DMARDs)
• Actemra^® (tocilizumab), a laboratory-made antibody that targets IL-6